Gene therapy is here - if you have $500,000

[IGotWorms 3,300]

https://www.google.com/amp/s/mobile.nytimes.com/2017/09/11/health/cost-gene-therapy-drugs.amp.html

 

The first gene therapy treatment in the United States was approved recently by the Food and Drug Administration, heralding a new era in medicine that is coming faster than most realize and that perhaps few can afford.

 

The treatment, Kymriah, made by Novartis, is spectacularly effective against a rare form of leukemia, bringing remissions when all conventional options have failed. It will cost $475,000.

 

With gene therapy, scientists seek to treat or prevent disease by modifying cellular DNA. Many such treatments are in the wings: There are 34 in the final stages of testing necessary for F.D.A. approval, and another 470 in initial clinical trials, according to the Alliance for Regenerative Medicine, an advocacy group.

 

The therapies are aimed at extremely rare diseases with few patients; most are meant to cure with a single injection or procedure. But the costs, like that of Kymriah, are expected to be astronomical, alarming medical researchers and economists.

 

One drug, to prevent blindness in those with a rare genetic disease, for example, is expected to cost between $700,000 and $900,000 per patient on average, noted Dr. Aaron Kesselheim, director of the program on regulation, therapeutics and law at Brigham and Womens Hospital.

 

Drug makers argue that the prices ought to reflect the value of a curative treatment to the patient. Dr. Kesselheim and other experts are far from convinced.

 

We dont pay the fire department that way, he said. When the fire department shows up at a burning house, they dont ask, How much is it worth to you to put out the fire?

More at the link...

[Cdub100 3,366]

This is great news. The price will come down in a few years.

[IGotWorms 3,300]

This is great news. The price will come down in a few years.

Not really, according to the rationales:

 

The reason why its a peculiar situation is not only because a lot of these gene therapy products are targeting small populations, said Matt Kapusto, chief executive of UniQure, which is developing hemophilia treatments. It is a one-time administration with potentially curative impact for the patient.

 

Mr. Kapusta also noted that one injection of UniQures drug could be expected to replace regular infusions of blood products that can cost $5 million over 10 years.

 

When you are spending a lot of money to develop therapies for a rare disease, you need to enable a larger price umbrella, Mr. Kapusta said. If you are saving $5 million per patient, that gives you a sense of value to the payer.

 

Jeffrey D. Marrazzo, chief executive of Spark Therapeutics, which is developing the drug to prevent a form of blindness, said it should be worth a lot to keep your eyesight. We should be compensated for generating that value, he said.

[vuduchile 1,940]

How much is my copay?

[fandandy 3,311]

 

Jeffrey D. Marrazzo, chief executive of Spark Therapeutics, which is developing the drug to prevent a form of blindness, said it should be worth a lot to keep your eyesight, adding; "Do you know how much contact solution is? I'm no economist but I imagine a person prolly spends damn near 20 Million dollars over the course of their life on contact solution alone, and that's just the solution. Those little plastic contact containers ain't exactly free either. Just sayin'."

 

[TD Ryan2 314]

I have one of those rare, genetic, eye disorders. My eye sight is slowly deteriorating... blind spots in my central vision that are growing slowly every year.

In my 30s, I could no longer track a golf ball.

In my 40s I can't play baseball catch with my son

Small, fast moving objects are nearly impossible for me to see.

50s? 60s?? 70s??? Not sure how long until I can no longer drive and then really "see".

So, for someone like me, this news is wonderful... and if I'm 70 and damn near blind and some big pharma CEO tells me that I spent 2mil on contacts and solution so now I can't have the cure, I will take him deep into the forest and jab his eyes out with a sharp stick... and they can lock my blind ass up for the 5 or 10 years I have left on this planet.

[IGotWorms 3,300]

I have one of those rare, genetic, eye disorders. My eye sight is slowly deteriorating... blind spots in my central vision that are growing slowly every year.

In my 30s, I could no longer track a golf ball.

In my 40s I can't play baseball catch with my son

Small, fast moving objects are nearly impossible for me to see.

50s? 60s?? 70s??? Not sure how long until I can no longer drive and then really "see".

So, for someone like me, this news is wonderful... and if I'm 70 and damn near blind and some big pharma CEO tells me that I spent 2mil on contacts and solution so now I can't have the cure, I will take him deep into the forest and jab his eyes out with a sharp stick... and they can lock my blind ass up for the 5 or 10 years I have left on this planet.

 

Frankly I think these people are scum.

 

But we need their cures so I'll guess we'll let em fock us all in the ass.

[Cdub100 3,366]

Not really, according to the rationales:

 

 

 

They are wrong. If it truly works the price will come down just like literally everything else.

[fandandy 3,311]

When this technology takes off those afflicted can fly overseas for treatment as well for much cheaper I'm sure. A flight to China is $1500? and I'm sure it won't cost no 500 grand in China.

[IGotWorms 3,300]

 

They are wrong. If it truly works the price will come down just like literally everything else.

Okay pal

[penultimatestraw 473]

 

Frankly I think these people are scum.

 

But we need their cures so I'll guess we'll let em fock us all in the ass.

It would be remotely justifiable if pharmaceutical companies were hurting financially. But they have profit margins among the highest in any industry.

 

Free market capitalism and medicine don't mix.

[penultimatestraw 473]

 

They are wrong. If it truly works the price will come down just like literally everything else.

It should, but what is a "fair" price for a life-saving drug? A cure for blindness?

[penultimatestraw 473]

I have one of those rare, genetic, eye disorders. My eye sight is slowly deteriorating... blind spots in my central vision that are growing slowly every year.

In my 30s, I could no longer track a golf ball.

In my 40s I can't play baseball catch with my son

Small, fast moving objects are nearly impossible for me to see.

50s? 60s?? 70s??? Not sure how long until I can no longer drive and then really "see".

So, for someone like me, this news is wonderful... and if I'm 70 and damn near blind and some big pharma CEO tells me that I spent 2mil on contacts and solution so now I can't have the cure, I will take him deep into the forest and jab his eyes out with a sharp stick... and they can lock my blind ass up for the 5 or 10 years I have left on this planet.

There is no cure for masturbation.

[titans&bucs&bearsohmy! 2,745]

When this technology takes off those afflicted can fly overseas for treatment as well for much cheaper I'm sure. A flight to China is $1500? and I'm sure it won't cost no 500 grand in China.

Why do we allow that?

 

Out tax dollars help find these cures. Then, we are priced out of them.

 

Reason #1,000,000 why our government is useless.

[penultimatestraw 473]

Why do we allow that?

 

Out tax dollars help find these cures. Then, we are priced out of them.

 

Reason #1,000,000 why our government is useless.

Allow what? Medical tourism? Pharmaceutical companies to charge whatever they think is appropriate?

[RaiderHaters Revenge 3,566]

they will just file it under "elective"

 

a dude wants to be a girl in the military we should pay for though

[titans&bucs&bearsohmy! 2,745]

Allow what? Medical tourism? Pharmaceutical companies to charge whatever they think is appropriate?

The latter. It is ridiculous that we pay substantially more than the rest of the world.

[penultimatestraw 473]

The latter. It is ridiculous that we pay substantially more than the rest of the world.

How do you fix that problem?

[titans&bucs&bearsohmy! 2,745]

How do you fix that problem?

I'd start by looking at how the rest of the world gets them cheaper.

[Baker Boy 1,477]

So are they passing on their old disgusting genes or their new super genes?

[penultimatestraw 473]

I'd start by looking at how the rest of the world gets them cheaper.

Cheap labor and less regulation of drug production (esp. patent law) overseas. Fragmented drug purchasing and greater profit incentive in the US.

[Baker Boy 1,477]

they will just file it under "elective"

 

a dude wants to be a girl in the military we should pay for though

But the dude does not become a girl, he becomes a dude with breast implants and no .

[edjr 5,547]

JCP has jeans for like 50, 60 bucks

[TD Ryan2 314]

there is no "free market" with pharmaceuticals and medical devices. Medicare, Medicaid, Insurers, Billing, Coding, and Policy makers all play an huge chess match with the pricing (not cost - PRICING) of all these things.

 

What's the price of the cure? Well... what's the "Average Selling Price" (ASP)?

The profit made will be based on the ASP and whatever reimbursement "bucket" the cure has been dumped in by Medicare/Medicaid and health policy makers... and the higher the ASP, the higher the reimbursement bucket, the more profit the doctor/hospital/care provider can make.

So the game for the Pharma executive is to make sure the ASP is as high as possible, to keep the product reimbursed as high as possible, to make sure the providers who use/apply the product can be profitable from using it.

 

See, if you cured cancer or blindness... but your cure/medicine's ASP was too low, then your reimbursement rate will be too low, then the Hospital CFO cannot justify purchasing your cure because it is a financial loss for the hospital. They'll opt to prescribe something more profitable that may or may not work... but makes everyone some money.

 

EDIT: the game gets even better... for many medicines/cures the health policy and reimbursement policies require that Federal Agencies (like the VA hospitals) receive the best, lowest prices on the medicine/cure... so, depending on the volume sold to the VA by the pharma companies, they will either make sure they charge the VA astronomical prices b/c the high volume sold there pushes the ASP really high... or... they will make a very cheap version of their product that, for some reason (i.e. size, regulation, etc) - is not available to the VA and therefore doesn't affect the ASP or their primary product.

[Cruzer 1,992]

I would like to see a price breakdown on this - see just what in the hell actually makes this cost $475K.

[penultimatestraw 473]

there is no "free market" with pharmaceuticals and medical devices. Medicare, Medicaid, Insurers, Billing, Coding, and Policy makers all play an huge chess match with the pricing (not cost - PRICING) of all these things.

 

What's the price of the cure? Well... what's the "Average Selling Price" (ASP)?

The profit made will be based on the ASP and whatever reimbursement "bucket" the cure has been dumped in by Medicare/Medicaid and health policy makers... and the higher the ASP, the higher the reimbursement bucket, the more profit the doctor/hospital/care provider can make.

So the game for the Pharma executive is to make sure the ASP is as high as possible, to keep the product reimbursed as high as possible, to make sure the providers who use/apply the product can be profitable from using it.

 

See, if you cured cancer or blindness... but your cure/medicine's ASP was too low, then your reimbursement rate will be too low, then the Hospital CFO cannot justify purchasing your cure because it is a financial loss for the hospital. They'll opt to prescribe something more profitable that may or may not work... but makes everyone some money.

 

EDIT: the game gets even better... for many medicines/cures the health policy and reimbursement policies require that Federal Agencies (like the VA hospitals) receive the best, lowest prices on the medicine/cure... so, depending on the volume sold to the VA by the pharma companies, they will either make sure they charge the VA astronomical prices b/c the high volume sold there pushes the ASP really high... or... they will make a very cheap version of their product that, for some reason (i.e. size, regulation, etc) - is not available to the VA and therefore doesn't affect the ASP or their primary product.

While the market isn't truly free, there definitely is more financial incentive for prescribing medicine in the US than overseas. Providers and insurers both game the system, but what they do pales in comparison to the pharmaceutical industry's pricing and marketing of drugs. Profitable drugs are heavily advertised and pushed on healthcare providers by glorified prostitutes/drug dealers, the pharmaceutical reps. Meanwhile, production of low price, often equally efficacious drugs is eternally inadequate to meet demand.

[IGotWorms 3,300]

I would like to see a price breakdown on this - see just what in the hell actually makes this cost $475K.

They tell you right from the horse's mouth that it doesn't cost that much. They don't set price based on cost. Apparently it's based on "value to the consumer" I.e. what is it worth to you to not have cancer?

 

Their other focked up rationale is that without the miracle cure you'd spend years going through expensive traditional therapy before you died. That would cost a fock ton of money. Now that they can cure you with one shot that shot should cost as much as all the therapy you would've gotten before because... well just because, they are horrible, greedy motherfockers who should be round up and shot, imo

[KSB2424 3,082]

Okay, couple things here.

 

1. Novartis is a Swiss Company. You know, Switzerland. Not a US based company.

 

2. How in the world can one read this news and not get excited at this medical breakthrough?

 

3. Nobody will have to pay 500K out of pocket, that is what insurance is for dummies. And insurance companies will negociate that price down probably in half, and be happy to pay it. Why? Because 250K is much cheaper than paying for months and years of other therapies, hospital stays, surgeries, etc. etc. A cure is most always cheaper than years and years of treatments.

 

4. And yes the Pharma company will have to make a profit to 1.) recoup the Billion dollars of R&D investment on this therapy plus the hunderds of others that failed and 2.) make a profit for their investors and shareholders so they can continue to thrive and create more breakthrough drugs.

 

5. And in 15 years it will go "Generic"and will be cheaper for the insurance providers.

 

Everybody wins, when Pharma creates new vaccines and new cures.

[IGotWorms 3,300]

http://www.npr.org/sections/health-shots/2017/09/11/550135932/r-d-costs-for-cancer-drugs-are-likely-much-less-than-industry-claims-study-finds

 

Cancer drugs cost far less to develop than industry-backed research asserts, an analysis published Monday asserts. Research and development costs are a major reason that drug companies justify high prices, so this dispute has a direct bearing on the cost of medical care.

 

The analysis, published in the current issue of JAMA Internal Medicine, concludes that it costs, on average, $650 million to develop a new cancer drug. The authors add in another $100 million or so to account for income those companies could have had if that money had been invested in the stock market instead of in new products.

 

That total is far lower than the $2.7 billion figure that the drug industry frequently points to when it justifies the soaring cost of medicine. (It's far higher than $320 million an inflation-adjusted figure from a 2001 study by the consumer group Public Citizen).

 

To arrive at this new figure, cancer physicians Vinay Prasad, at Oregon Health and Science University, and Sham Mailankody, at the Memorial Sloan Kettering Cancer Center, took a novel approach. They identified 10 companies that each had a single cancer drug on the market. They looked up the companies' research and development costs, as reported in their federal stock reporting paperwork, to come up with the average figure of $650 million.

 

The companies reaped substantial rewards. On average, the study found each product produced seven times as much revenue as it cost in research and development and the drugs will yield profits for years to come.

 

"I think these results would suggest that pharmaceutical drug development is extremely lucrative and the current drug prices are not necessarily justified by the R & D [research and development] spending on these drugs," Mailankody says.

[Bert 1,087]

It should, but what is a "fair" price for a life-saving drug? A cure for blindness?

True but the bigger issue in "fair" is how do you pay for the billions spent on development? How do you pay for the R&D for things that don't work but lead to the success of other products?

 

It is not a simple solution.

[IGotWorms 3,300]

Okay, couple things here.

 

1. Novartis is a Swiss Company. You know, Switzerland. Not a US based company.

 

2. How in the world can one read this news and not get excited at this medical breakthrough?

 

3. Nobody will have to pay 500K out of pocket, that is what insurance is for dummies. And insurance companies will negociate that price down probably in half, and be happy to pay it. Why? Because 250K is much cheaper than paying for months and years of other therapies, hospital stays, surgeries, etc. etc. A cure is most always cheaper than years and years of treatments.

 

4. And yes the Pharma company will have to make a profit to 1.) recoup the Billion dollars of R&D investment on this therapy plus the hunderds of others that failed and 2.) make a profit for their investors and shareholders so they can continue to thrive and create more breakthrough drugs.

 

5. And in 15 years it will go "Generic"and will be cheaper for the insurance providers.

 

Everybody wins, when Pharma creates new vaccines and new cures.

I appreciate your perspective KSB. It's complete party line for the drug companies, but you do have some good points there.

 

Namely, that there needs to be financial incentive for new cures and therapies. Certainly under our current system. And one legitimate concern, imo, is that if we went with a national healthcare system or the like you'd then remove a great deal of incentive for innovation.

 

But where does that cross into massive profiteering off of the sick and vulnerable? Because it sure sounds to me like that happens quite a bit. Which is probably just as bad, if not worse, than war profiteering.

 

Yes you need to make a profit. Yes you can make a huge profit -- greater than just about any other sector out there -- because people NEED your product just to live. No, that doesn't make obscene profiteering right.

[KSB2424 3,082]

I would like to see a price breakdown on this - see just what in the hell actually makes this cost $475K.

 

It's not like putting a cost breakdown on a widget. You don't say, the tires cost 10, the body costs 20 and the engine costs 30 so it cost 60 so we'll sell it for 90.

 

There is sunk costs from the many other failed R&D on things that never make it to market. The overwhelming majority of R&D never meets the light of day, but it all still costs money. That is included in the complex pricing models of drugs, vaccines and cures. I

 

Worms's link is one study where they say this sunk costs is overstated. Others disagree, but if so that is wrong, so I appreciate that link.

 

Either way, nobody pays 500K out of pocket. You only pay what your insurance max deems it to be. And that is usually 5 figures.

[titans&bucs&bearsohmy! 2,745]

I appreciate your perspective KSB. It's complete party line for the drug companies, but you do have some good points there.

 

Namely, that there needs to be financial incentive for new cures and therapies. Certainly under our current system. And one legitimate concern, imo, is that if we went with a national healthcare system or the like you'd then remove a great deal of incentive for innovation.

 

But where does that cross into massive profiteering off of the sick and vulnerable? Because it sure sounds to me like that happens quite a bit. Which is probably just as bad, if not worse, than war profiteering.

 

Yes you need to make a profit. Yes you can make a huge profit -- greater than just about any other sector out there -- because people NEED your product just to live. No, that doesn't make obscene profiteering right.

If you need a financial incentive to cure cancer, you are the definition of lowlife piece of sh!t.

[penultimatestraw 473]

Okay, couple things here.

 

1. Novartis is a Swiss Company. You know, Switzerland. Not a US based company.

 

2. How in the world can one read this news and not get excited at this medical breakthrough?

 

3. Nobody will have to pay 500K out of pocket, that is what insurance is for dummies. And insurance companies will negociate that price down probably in half, and be happy to pay it. Why? Because 250K is much cheaper than paying for months and years of other therapies, hospital stays, surgeries, etc. etc. A cure is most always cheaper than years and years of treatments.

 

4. And yes the Pharma company will have to make a profit to 1.) recoup the Billion dollars of R&D investment on this therapy plus the hunderds of others that failed and 2.) make a profit for their investors and shareholders so they can continue to thrive and create more breakthrough drugs.

 

5. And in 15 years it will go "Generic"and will be cheaper for the insurance providers.

 

Everybody wins, when Pharma creates new vaccines and new cures.

 

1. It doesn't matter where the drug is developed, if the companies are paid in dollars for their drugs.

 

2. Gene therapy is a good thing, but we also have horrifically overpriced healthcare, and adding bazillion dollar drugs ain't gonna reduce that cost.

 

3. For a cure for cancer, there may be a net savings on expenditures, with profit shifting from healthcare providers to drug manufacturers - a worthwhile trade off. But nobody is racking up a huge medical bill for blindness, though the impact on quality of life is immeasurable.

 

4. Drug companies already make obscene profits, greater than almost any industry. The R&D costs are conveniently overestimated to justify their bloated pricing.

 

5. When the drug goes off patent, a new "me too" drug will take its place, while production of the original drug is reduced.

 

Nobody wants the pharmaceutical industry to stop being innovative. We just want their ethics to exceed their greed.

[penultimatestraw 473]

True but the bigger issue in "fair" is how do you pay for the billions spent on development? How do you pay for the R&D for things that don't work but lead to the success of other products?

 

It is not a simple solution.

Of course it isn't simple, but the pendulum has swung too far in favor of profit over affordable treatment.

[Bert 1,087]

Of course it isn't simple, but the pendulum has swung too far in favor of profit over affordable treatment.

Surgery, medical procedures, hospital stays, any thing that involves seeing a doctor I absolutely agree.

 

The drug issue I mostly agree but it is easier to understand given the risk and burn rate on cash that these companies must deal with before they produce a marketable drug.

[TD Ryan2 314]

 

The drug issue I mostly agree but it is easier to understand given the risk and burn rate on cash that these companies must deal with before they produce a marketable drug.

 

understandable.

the argument that, "it only costs $1 to make" is rubbish... long before efficient, $1 production was an option, millions went into R&D, etc. But, it was mentioned above, sometimes that R&D is funded by public money.

I don't know what the answer is... it's messy ground once you start trying to tell companies what to charge for their product/service... but there is a LOT of corporate greed/gluttony in the pharma industry... I see it in the Boston area... something's gotta' give.

[IGotWorms 3,300]

If you need a financial incentive to cure cancer, you are the definition of lowlife piece of sh!t.

Well I don't think it's quite that simple. A lot of money and resources goes into it so there does need to be a payoff in the end. That's just human nature or, at the very least, the way our capitalist system works.

 

But there is a line, and I think these focks have gone way past it.

 

I actually really like that Martin Shrkeli guy because I think he exposed the entire industry in a visceral way. He may be the poster child for pharmaceutical greed and excess but he's a pretty good representative of the industry as a whole.

[Thornton Melon 542]

I would like to see a price breakdown on this - see just what in the hell actually makes this cost $475K.

 

It typically costs in the hundreds of millions to develop a new cancer drug.

 

I work in biopharmaceutical manufacturing, and I know it costs alot to manufacture these types of drugs. Materials, equipment, components - everything costs a sh!tload of $$ because of the stringent technical requirements, documentation, and regulations.

 

These new gene therapies are probably even more costly to manufacture than typical biotech drugs because of the technological hurdles. They use viruses to deliver the genes to the patients cells. In typical biopharmaceutical manufacturing, viruses are the enemy, and must be filtered out of the final product. The trick with gene therapies is to be able to filter out the unwanted viruses while letting the right ones through.

 

Is $475K too much for this drug? Probably, but one resource that I read thought it would be higher. Do pharma companies make obscene profits by pushing their products? Yeah. Do they need to make huge profits to re-invest in research? Yeah. Does my CEO need to make a $25M bonus every year? Hell, no.

[Bert 1,087]

Nobody wants the pharmaceutical industry to stop being innovative. We just want their ethics to exceed their greed.

Same thing with surgeons, doctors and anesthesiologists. They are just as bad if not worse.